Gene Therapy: Let’s Talk about Safety, Baby

Let’s talk about all the good things and all the bad things that may be when it comes to realizing gene therapy’s promise. While only 19 gene therapies are approved globally, more than 2,000 are in development, making it one of the most burgeoning areas in medicine, promising functional cures. Tempering this excitement are safety concerns – with several clinical holds due to patient deaths. Further, the inability to re-dose AAVs limits the long-term durability of treatment. In response, companies are solving these challenges with new technologies promising safety and re-dosability. They are also sharing data, which can catch early safety signals when trial sizes are small. In an interactive discussion, experts will weigh four areas that hold promise in advancing safety and re-dosability of gene therapy: new delivery technologies; new vectors; nonviral approaches; and safety data sharing. This discussion is increasingly important in a $6B+ gene therapy market expected to hit $42B+ by 2030.